| 发明名称 |
COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING |
| 摘要 |
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy. |
| 申请公布号 |
EP2644700(A1) |
申请公布日期 |
2013.10.02 |
| 申请号 |
EP20130165321 |
申请日期 |
2006.06.23 |
| 申请人 |
ISIS PHARMACEUTICALS, INC.;COLD SPRING HARBOR LABORATORY |
发明人 |
BAKER, BRENDA F.;KRAINER, ADRIAN R.;HUA, YIMIN |
| 分类号 |
C12N15/113;A61K31/7115;A61K31/712;A61K31/7125;C12N15/11 |
主分类号 |
C12N15/113 |
| 代理机构 |
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